Ontology highlight
ABSTRACT:
SUBMITTER: Wong CH
PROVIDER: S-EPMC4015652 | biostudies-literature | 2014 Apr
REPOSITORIES: biostudies-literature
Wong Chun-Ho CH Nguyen Lien L Peh Jessie J Luu Long M LM Sanchez Jeannette S JS Richardson Stacie L SL Tuccinardi Tiziano T Tsoi Ho H Chan Wood Yee WY Chan H Y Edwin HY Baranger Anne M AM Hergenrother Paul J PJ Zimmerman Steven C SC
Journal of the American Chemical Society 20140422 17
A working hypothesis for the pathogenesis of myotonic dystrophy type 1 (DM1) involves the aberrant sequestration of an alternative splicing regulator, MBNL1, by expanded CUG repeats, r(CUG)(exp). It has been suggested that a reversal of the myotonia and potentially other symptoms of the DM1 disease can be achieved by inhibiting the toxic MBNL1-r(CUG)(exp) interaction. Using rational design, we discovered an RNA-groove binding inhibitor (ligand 3) that contains two triaminotriazine units connecte ...[more]